Hematologic malignancies (a.k.a., blood cancer or liquid tumors) are forms of cancer that begin in the cells of blood-forming tissue, for example, the bone marrow or in the cells of the immune system. Examples of hematologic cancers include acute and chronic leukemia, lymphomas, multiple myeloma and myelodysplastic syndromes.
Leukemia is a type of cancer that occurs in the bone marrow and in the blood. There are two types of leukemia: Lymphocytic leukemia involves lymphocytes. Myelogenous leukemia involves granulocytes. These white blood cells are important in fight infections. Lymphoma is a type of cancer that develops in the lymphatic system. There are two general types of lymphoma depending on how cancer spreads. In Hodgkin lymphoma, the cancer spreads from one group of lymph nodes to another in a certain order. In non-Hodgkin lymphoma, the cancer spreads from one group of lymph nodes to another in a random order. Myeloma is a cancer that causes the plasma cells to form a tumor in the bone marrow. Myeloma is usually found in multiple places in the body, thus often called multiple myeloma.
Treatment of hematologic malignancies includes “watchful waiting” (e.g., in chronic lymphoid leukemia or CLL) or symptomatic treatment (e.g., blood transfusions in myelodysplastic syndromes or MDS). More aggressive forms of disease require treatment with chemotherapy, radiotherapy, immunotherapy or a bone marrow transplant. For example, rituximab, a chimeric monoclonal antibody against the protein CD20, is used to treat B-cell-derived hematologic malignancies, including follicular lymphoma and diffuse large B-cell lymphoma.
Isocitrate dehydrogenases are metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies, including acute myelogenous leukemia and glioma, a type of aggressive brain tumor with poor prognosis. Normally, IDH enzymes help to break down nutrients and generate energy for cells. Mutations in the IDH genes are strongly correlated with the development of acute myelogenous leukemia, glioma, chondrosarcoma, intrahepatic cholangiocarcinoma, and angioimmunoblastic T-cell lymphoma cancers. They also cause D-2-hydroxyglutaric aciduria and Ollier and Maffucci syndromes.
Hematologic and solid malignancies are placing an increasing burden on society, impairing the health and lives of those affected. Although medications have been developed to treat some of these diseases and conditions, the available treatments are often limited in terms of clinical effectiveness and at the same time have undesirable side effects.
There is an urgent and growing need for innovative therapeutics and treatment methods that provide improved clinical effectiveness with reduced side effects.